ATALUREN - confused about the information given from PTC Therapeutics and Genzyme

Can someone PLEASE help me understand the information given from PTC Therapeutics and Genzyme, regards to Ataluren. Our son can use
Ataluren, and he is about to stop walking. He didn’t participate in the
previous clinical trials, because the enrollment was closed before our son
could enter the trial. I know there are many other boys ”out there” in the same
position.

I am asking for several other parents coming from Norway,but also parents coming from other contries.

First we had this article from the Annual Meeting of the American Academy of Neurology, published

April 13th. This article didn’t say anything that this is going to be a follow-on clinical study of Ataluren, so I thought ohhhhh finally
Ataluren will be on the market in the USA (if approved by the FDA):

Based on these results, PTC Therapeutics, the company that developed ataluren, is planning to seek approval for its
use in DMD from the United States Food and Drug Administration. Because of
the mechanism by which the drug overcomes the effects of the mutation, it would
be appropriate for only about 13% of DMD patients, according to study presenter
Ted Abresch, Research Associate in Neuromuscular Disease at the University of
California at Davis.

Then today, this article was publised by Genzyme. They are talking about a follow-on open label clinical study for previous trial
participants, in Europe.

Ataluren update from Genzyme

Follow-on open label clinical study announced for previous trial participants

As part of our continuing assessment, Genzyme plans to initiate a follow-on clinical study of ataluren in nmDBMD patients who
previously participated in the clinical trials in Europe, Israel and Australia.
All patients who have previously participated in the clinical trials with
ataluren in nmDBMD, irrespective of their current clinical status, will be
eligible to participate.

We believe that this clinical study will allow for the collection of additional information on ataluren in nmDBMD while providing access
to ataluren to all patients who have been involved in earlier clinical trials.
Currently we are working to develop the timelines and design for this clinical
study and we plan to update the community, with these additional details in the
very near future. We are reaching out to the original trial investigators to
make them aware of this new development and they may be in touch with eligible
families directly as well.

Replies to This Discussion

In theory, IF FDA approves ataluren, it COULD be available to all boys with nonsense mutations. But we need to keep in mind that nothing is simple and that sometimes drugs are sometimes approved for a certain group of individuals and not another under certain conditions (conditional approval) We have no information about what will likely happen, but sometimes FDA grants a conditional approval, setting certain conditions for using the drug and potentially recommending additional studies to understand or better understand the drug's safety of efficacy.

With regard to EMA, things are a bit different, each country having specific regulatory requirements as well as laws related to access and reimbursement.

Being in the DMD-community for almost 9 years now, I certainly know nothing is simple. Its a roller coaster turning from hope one day, and to disappointment the next day. We just have to deal with it, and try to stay positive to our boys. Its hard though.

Just one more question; Do you know if PTC Therapeutics applied to FDA for a certain group (conditional approval), or not ?

I read the translation from our parentproject from Action Duchenne conference London in the end of last yaer. Mr. Peltz said there that PTC will try to get approval for Ataluren in July this year. So I hope that will fill NDA in this month because they got fast track for dystrofinopathy. And if I understood this well they can fill it in phase 3 after 6 month of clinical trial.

I trying to get some information from internet but nothing...

Our son is in the open label study but I don't know very much other than that. It is 36 weeks, visits every 12 weeks, low dose, of course :) Don't know what happens after the 36 weeks.